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Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses
Azam Safary1,2, Hakimeh Moghaddas-Sani3, Mostafa Akbarzadeh-Khiavi4
1Connective Tissue Diseases Research Center, Tabriz University of Medical Sciences, Tabriz, Iran.
Expert Opinion on Biological Therapy
|March 3, 2021
Summary
Enzyme replacement therapy (ERT) for mucopolysaccharidoses (MPS) has limitations. Combination approaches, including stem cell or gene therapy, offer promising personalized treatments for these inherited lysosomal storage disorders.
Area of Science:
- Biochemistry
- Genetics
- Medical Genetics
Background:
- Mucopolysaccharidoses (MPS) are inherited lysosomal storage disorders (LSDs) with diverse clinical features, including skeletal and neurological issues.
- Current enzyme replacement therapy (ERT) for MPS faces challenges such as limited enzyme distribution, immune reactions, and high costs.
Purpose of the Study:
- To review ERT-based combination therapies for MPS.
- To discuss recent advancements and future strategies for MPS treatment.
Main Methods:
- Comprehensive literature review of published studies on PubMed and Scopus.
Main Results:
- ERT-based combination therapies, particularly with stem cell or gene therapy, show potential for personalized treatment of MPS.
- These combined approaches may enhance therapeutic responses and minimize side effects compared to ERT alone.
Conclusions:
- Combination therapies are crucial for addressing the complex pathophysiology of MPS.
- Future strategies should explore novel drug combinations targeting secondary pathological pathways for improved MPS management.
Keywords:
Combination therapyenzyme replacement therapylysosomal storage disordersmucopolysaccharidosisMore Related Videos
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